FOXG1 disorder
MONDO:0100040A monogenic disease that has material basis in mutation in the FOXG1 gene.
Also known as: FOXG1 disorder, FOXG1 inherited genetic disease, FOXG1 syndrome, FOXG1 syndrome due to intragenic alteration, FOXG1-related epileptic-dyskinetic encephalopathy, Rett syndrome, congenital variant, inherited genetic disease caused by mutation in FOXG1
3 clinical trials for this condition and its sub-types, 2 tagged with FOXG1 disorder itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →-
Gene therapy breakthrough? new trial targets rare brain disorder FOXG1
Disease control By invitation onlyThis early-stage trial tests a new gene therapy called FRF-001 for FOXG1 syndrome, a rare genetic disorder that affects brain development. The therapy is given as a single injection into the brain's fluid spaces. The study will include 12 children and adults and will check for sa…
Phase 1/2 • Sponsor: FOXG1 Research Foundation • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC
-
New program aims to ease burden on families of kids with rare diseases
Symptom relief By invitation onlyThis study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare …
Sponsor: Children's National Research Institute • Aim: Symptom relief
Last updated Jun 27, 2026 09:00 UTC